1. Joint BES/BBS Seminar Patient Preference Studies – Introduction
Conny Berlin
Global Head Quantitative Safety & Epidemiology, Novartis
IMI PREFER Project Leader
Basel, 22 November 2018

2. Disclaimer
The views and opinions expressed in this presentation are those of the presenter and do not necessarily reflect the official policy or position of Novartis or any of its officers.

3. Patient Preference Studies
Background & current environment
What are patient preferences?
What are patient preference sensitive decisions?
What patient preference methods do exist?
What are open questions and how do we get answers?
Contacts & references

4. Background & current environment
FDA to release 4 guidelines on patient-focused drug development between ( )
EMA includes patient involvement (and patient preference studies) in CHMP work plan 2017/2018
New EFPIA guideline on working with patient groups
IMI «PREFER» ( )
IMI «PARADIGM» ( )
ICH guideline includes advice on how patient input can contribute to Clinical Overview benefit-risk assessment
... Driving for more patient engagement
... Specifically FDA: Incorporation of Patient’s Voice in Drug Development and Decision-Making; systematic collection and analysis of patient experience data (from anecdotes to systematic, reproducible, generalizable…. Information)
“Roadmap” for Patient Engagement in R&D has been defined and published
Additional “Roadmap” areas, e.g. Patient Access, are currently being developed
First ever book of its kind published
Why, what, how and when of Patient Engagement in HTA decision making

5. New Checklist utilized by FDA to evaluate submitted Patient Experience data
EXAMPLE
FDA has begun documenting what patient experience data have been submitted with an application through a new "Patient Experience Data" section in clinical review documentation
We might expect FDA to use this approach consistently and data may be included in the label, i.e. be promotable
Source:

6. New Checklist utilized by FDA to evaluate submitted Patient Experience data
EXAMPLE
FDA has begun documenting what patient experience data have been submitted with an application through a new "Patient Experience Data" section in clinical review documentation
We might expect FDA to use this approach consistently and data may be included in the label, i.e. be promotable
Source:

7. What are patient preferences*?
Measures of the relative importance of benefits, harms and other characteristics of treatments Patient preference are the basis of how patients choose a particular treatment over other available options.
*Definition per Medical Device Innovation Consortium (MDIC) Patient-Centered Benefit-Risk

8. Three types of patient preferences
What it Measures
Attributes
What Matters
Relative Importance
How much it matters
Tradeoffs
What tradeoffs patients are willing to make between benefits, harms, and other aspects
It covers the use of patient preferences for benefit-risk and reimbursement decisions but also the use of preferences to inform development programs or to show that there is an unmet need; e.g. what are relevant endpoints for patients which should be measured in clnical trials?
The objetcive / purpose of a preference study should always be part of the discussion and clearly described
Today’s case study presentations will show how preferences have either been used or are intended to be used.
Adapted from RTI-HS

9. Which treatment is best?
Reduction in days hospitalized (benefit)
Probability of infection (risk)
Ideal D
Device C is superior on both benefit and risk
Preference information is not needed to determine the best treatment
Source: MDIC Patient Centered Benefit-Risk Framework Report Public Release, May 13,2015

10. Now which treatment is best?
Preference information is needed to choose between devices A and C B E C D A
Reduction in days hospitalized (benefit)
Probability of infection (risk)
Ideal
Source: MDIC Patient Centered Benefit-Risk Framework Report Public Release, May 13,2015

11. Patient preference sensitive decisions
.. related to treatment options: 1. multiple treatment options exist and there is no option that is clearly superior for all patients 2. when the evidence supporting one option over others is considerably uncertain or variable and/or 3. patients’ views about the most important benefits and acceptable risks of a treatment vary considerably within a population, or differ from those of healthcare professionals.

12. Patient preference methods
Type
What it Measures
Methods
Attributes
What Matters
Qualitative or simple quantitative methods
What: Can often be obtained using qualitative methods; Simple quantitative methods can be used to separate those attributes that matter to patients from those attributes that do not
Adapted from RTI-HS; Brett Hauber, ICPE 2018, Prague

13. Patient preference methods
Type
What it Measures
Methods
Attributes
What Matters
Qualitative or simple quantitative methods
Relative Importance
How much it matters
Quantitative methods
What: Can often be obtained using qualitative methods; Simple quantitative methods can be used to separate those attributes that matter to patients from those attributes that do not
Relative importance: Requires using quantitative methods that provide a weight for each attribute
Adapted from RTI-HS; Brett Hauber, ICPE 2018, Prague

14. Patient preference methods
Type
What it Measures
Methods
Attributes
What Matters
Qualitative or simple quantitative methods
Relative Importance
How much it matters
Quantitative methods
Tradeoffs
What tradeoffs patients are willing to make between benefits, harms, and other aspects
Simple choices by patient, approximate or accurate quantitative methods
What: Can often be obtained using qualitative methods; Simple quantitative methods can be used to separate those attributes that matter to patients from those attributes that do not
Relative importance: Requires using quantitative methods that provide a weight for each attribute
Tradeoffs:
1. Patients are willing to make to obtain or avoid a given attribute.
2. Can be approximated by comparing the weights that patients assign to each attribute
3. Obtaining accurate trade-off information may require quantitative methods designed explicitly for this purpose
Adapted from RTI-HS; Brett Hauber, ICPE 2018, Prague

15. Patient preference methods
Type
What it Measures
Methods
Attributes
What Matters
Qualitative or simple quantitative methods
Relative Importance
How much it matters
Quantitative methods
Tradeoffs
What tradeoffs patients are willing to make between benefits, harms, and other aspects
Simple choices by patient, approximate or accurate quantitative methods
Complexity
What: Can often be obtained using qualitative methods; Simple quantitative methods can be used to separate those attributes that matter to patients from those attributes that do not
Relative importance: Requires using quantitative methods that provide a weight for each attribute
Tradeoffs:
1. Patients are willing to make to obtain or avoid a given attribute.
2. Can be approximated by comparing the weights that patients assign to each attribute
3. Obtaining accurate trade-off information may require quantitative methods designed explicitly for this purpose
Adapted from RTI-HS; Brett Hauber, ICPE 2018, Prague

16. Patient preference methods
Most recent categorization by IMI PREFER

17. Open questions in patient preference studies
Whose preferences?
Cognitive requirements?
Which methods?
How relate to trial endpoints?
Validity?
Which patients?
How communicate?
When should we do it?
Roles in ED?
Expertise needed?
Sample size?
Can patients do these studies?
Roles in FD?
Payer requirements?
Many open questions which need answers to make sure that preference studies are done in a scientific rigor way and provide valid and reliable information for decision makers in industry, HAs and HTA bodies.
Some of the questions will be touched in today’s presentations.
Many of the questions are currently under consideration in IMI PREFER.
Regulatory requirements?
Roles in reimbursement?
Cost?
Preference-based subgroups?
Industry / regulatory /payer collaboration?
Time?
Stability?
Do we really need it?
Source: Bennett Levitan (Johnson&Johnson)

18. Goal of the IMI PREFER Project
By developing expert and evidence-based recommendations, PREFER aims to guide industry, regulatory authorities and HTA bodies and reimbursement agencies on how patient preferences can be assessed and used to inform medical product decision making.
Medical product decision making
DEV: BR
Patient access / HEOR: HTA
PP studies which might impact both decisions
Axel M: HTA as specific example
5 year project:

19. 33 PREFER partners

20. Who you can contact in your companies (in Basel)
Novartis: Conny Berlin Sheila Dickinson Roche: Marloes van Bruggen Actelion: Monika Brand

21. (a few) References http://www.imi-prefer.eu/publications/
MDIC Patient Centered Benefit-Risk Framework Report Public Release, May 13,
PREFER webinar: Eliciting Patients’ Preferences for Parkinson’s Devices: